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Seems like nothings happening....

Maybe its the activity level on this site but it seems like nothing is happening... I almost get the feeling no one sees a cure any more... I find myself starting to lean that way. Seems like theres not even good news to post about.... its getting sad...

Here is an update from Wise dated 12-15-15. I also have another one to pass along as well.

DaDutchman,

We did have some trouble finding patients that were AIS A chronic in Hong Kong for our phase I trial. However, in China, this was not as much of a problem. So, in the upcoming Phase II trial that will be submitted to the U.S. Food and Drug Administration (FDA) and to the Drug Controller General of India (DCGI), we have decided to include patients who were initially AIS A and then converted to B or C. We call these severe stable spinal cord injury rather than complete spinal cord injury who are not walking. Note that most patients that are initially AIS B or C generally recover walking within a year. As soon as the FDA and the DCGI approve the protocol, we will of course announce the trial on www.clinicaltrials.gov and on this site, along with all the contact information. The participating centers will be the ones that choose people for the trial. Each of 3 centers in each country will choose 6 participants in the trial.

The main reason for the delay is the long time it has taken for us to prepare the IND (the initial new drug/device) application to the US FDA and India's DCGI for the clinical trials. The document is extensive (over 1000 pages) and requires detailed information and tests concerning the manufacturing of the cells, the shipping, and the transplantation, as well as all the animal and safety studies. We have scheduled a pre-IND meeting with the US FDA in January and hope to submit the final application shortly after that meeting. Likewise, the clinical trial is being evaluated by the institutional review boards (IRB) in India and will be submitted to the DCGI as soon as the IRB applications have been approved. We will then work on the applications for the European Medicine Agency (EMA) and Norway as well as the China Food and Drug Administration (CFDA).

Three other tasks must be accomplished before we can start the trials. The first is money of course. I have been spending a lot of time raising money for the trials. The fact that we are going forward with the US FDA and the DCGI applications indicate that we are confident that we have the funds of the trial in the U.S. and India. The second is the walking programs. We have also been spending a lot of time training the various centers to do the intensive locomotor training. The Kunming walking program is quite different from other programs around the world. Several years ago, I took groups of American and Norwegian doctors to Kunming to see the walking program. Last August, we have already taken a group of Indian doctors to the Kunming Tongren Hospital to see the walking program and surgery. The third is the cell processing. We will need to certify each hospital for their ability to handle the cells that will be shipped to them. This will require testing and validation of the hospitals. Before we did the trials in China, we tested over 100 units of umbilical cord blood to make sure that the transplanted cells are sterile, alive, and able to grow.

The phase II trials that we are planning to carry out in India and the U.S. will transplant 6.4 million HLA-matched umbilical cord blood mononuclear cells (UCBMNC) into the spinal cords of all the patients and then randomize them to either a 6-week course of lithium carbonate or a 6-week course of lithium. The inclusion criteria are C5-T11, severe and stable SCI. The goals of the trial are to 1) to determine the feasibility of doing the 6-hours a day, 6-days a week, for 6 month walking training in these countries, 2) to determine that a 6-week course of lithium is beneficial when combined with UCBMNC transplants, and 3) to confirm the results from Kunming. In India, if the trial is successful, we hope that we will get conditional marketing approval so that the therapy will be available at cost to qualified doctors and their patients. In the U.S. and EMA, we hope that the FDA will grant compassionate use approval for the treatment for individual patients and qualified doctors.

If the phase II trial show that lithium is not beneficial when combined with UCBMNC transplants, we will omit lithium from the phase III trial, which will compare UCBMNC transplants or no transplants. Of course, if lithium turns out to be beneficial when combined with UCBMNC transplants, the phase III trial will include lithium and compare surgery alone, surgery plus UCBMNC, surgery plus lithium, and surgery plus UCMBNC and lithium. As pointed out above, if the phase II trials confirm the efficacy of UCBMNC (>50% recovery of walking, bowel, and bladder function), we expect the DCGI to give conditional market approval and the U.S. FDA to approve compassionate use of UCBMNC transplants by qualified doctors and patients.

It is getting to beyond the two-year mark for some parents in the original Kunming study. We are currently trying to find a time and situation to bring the patients back. It is a lot of trouble and costly for patients to travel back to the center. So, we need to raise the funds for that as well. Several have come back on their own to visit because they live nearby. At the present, Kunming is doing another trial... assessing the effects of surgery and walking training alone, without cell transplants.

Regarding participation in an upcoming trial in China, several changes this year may postpone the trial. Last April, the China Food and Drug Administration announced new regulations that seemed to require a two-stage approval for clinical trials, one at the provincial level and a final approval in Beijing. However, they apparently did not have the infrastructure to implement this two-stage approval. In August, we heard that they were going to change the system to just provincial approval of selected hospitals as "stem cell centers" and allow trials as such centers. Three of our centers have applied for stem cell center status. So, we are waiting for these applications to clear before going forward.

The path for getting national approval of a cellular therapy is still unclear. I assume that the original proposal for initial provincial approval followed by Beijing approval still stands. No non-Chinese company is allowed to do stem cell therapies. So, whatever company wants to do a trial in China must find a Chinese company to partner with. Until the recent stock market crash, some Chinese investment groups were actually looking around for American stem cell companies to acquire. Right now, things are a bit mixed up in terms of clinical trials in China. I hope that it will clear up soon. As soon as we know that a trial has been approved, it will be announced here on CareCure and also on www.clinicaltrials.gov.

We are applying for the trials in the U.S. and India. Those applications are still in progress. As soon as something solid is know, we will likewise announce these trials here and on www.clinicaltrials.gov. None of the trials are recruiting and so there is no point in applying or trying to get on various lists.

I'm pinning my hopes on other solutions. I would love for this to work - but they have never told us about anything where we get sensation back.
All we hear about is some motor control that isn't really useful.
I know its a sort of progress, but for people hoping of better we need progress beyond this - or at least a description of what will happen.

If after 2 years people may or may not have good benefits but we don't know seems like a problem. Whats the point of moving ahead with more trials if you don't know the long term results of the first trial?

I'm pinning my hopes on other solutions. I would love for this to work - but they have never told us about anything where we get sensation back.
All we hear about is some motor control that isn't really useful.
I know it's a sort of progress, but for people hoping of better we need progress beyond this - or at least a description of what will happen.

If after 2 years people may or may not have good benefits but we don't know seems like a problem. Whats the point of moving ahead with more trials if you don't know the long term results of the first trial?

Surely it would be best to get this information?

Welcome PBR, you should tell us about yourself.

I was injured 20 years ago, C5/6 incomplete.

In the ChinaSCINet Phase II trial, return of function was much more extensive than just motor control. 75% of the participants regained walking with minimal assistance and more than half recovered bowel and bladder function. Many of these patients went from complete to incomplete injuries which enabled them to become much more independant. Plans are underway to bring these patients back for a two year evaluation. We are working hard to do the trial in the US, India, China, and hopefully Norway.

Please excuse my ignorance but I've not been actively following the CC. I do read the Rutgers' newsletters and are we still awaiting publication? It's strange - a lot of the people used to post no longer posting. Is Dr. Young taking a hiatus from posting on his forum?